Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of...

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Hlavní autoři: Richard S. Finkel, Eugenio Mercuri, Basil T. Darras, Anne M. Connolly, Nancy L. Kuntz, Janbernd Kirschner, Claudia A. Chiriboga, Kayoko Saito, Laurent Servais, Eduardo F. Tizzano, Haluk Topaloğlu, M. Tulinius, Jacqueline Montes, Allan M. Glanzman, Kathie M. Bishop, Zhensbao Zhong, Sarah Gheuens, C. Frank Bennett, Eugene Schneider, Wildon Farwell, Darryl C. De Vivo
Médium: Artigo
Jazyk:angličtina
Vydáno: 2017
On-line přístup:https://doi.org/10.1056/nejmoa1702752
https://www.nejm.org/doi/pdf/10.1056/NEJMoa1702752?articleTools=true
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https://doi.org/10.1056/nejmoa1702752
https://www.nejm.org/doi/pdf/10.1056/NEJMoa1702752?articleTools=true

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